Hypereosinophilic SyndromePhase 2NCT02101138
Study to Evaluate Safety and Efficacy of Dexpramipexole (KNS-760704) in Subjects With Hypereosinophilic Syndrome
A Phase 2 study testing Dexpramipexole in people with Hypereosinophilic Syndrome, run by National Institute of Allergy and Infectious Diseases (NIAID) at 1 site in United States.
Trial summary
Background: Eosinophils are white blood cells that fight infections. In people with hypereosinophilic syndrome (HES), eosinophil levels are too high and can damage their organs. HES is usually treated with steroids, but steroids can cause side effects and stop working over time. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Hypereosinophilic Syndrome
- What is being tested?
- Dexpramipexole
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 1 siteUnited States
- Study dates
- 14 Mar 2014to 23 Oct 2019
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- A subject will be eligible for participation in the study only if all of the following criteria apply:
- The subject is male or female, age greater than or equal to 18 years
- The subject has a documented history of HES requiring greater than or equal to 10 mg prednisone (or equivalent) to maintain disease control.
- HES is defined as 1) peripheral blood eosinophilia (>1500 eosinophils/microL) on at least two occasions, 2) signs and symptoms of organ system involvement attributable to the eosinophilia, and 3) exclusion of secondary causes of eosinophilia, such as parasitic helminth infection, drug hypersensitivity and neoplasms, for which appropriate therapy is directed at the underlying cause
- HES symptoms are stable on the current corticosteroid dose.
- The subject agrees to storage of samples for study.
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- Females are eligible for this study if they are:
- of non-childbearing potential (i.e., women who have had a hysterectomy or tubal ligation or are postmenopausal as defined by no menses in 1 year); OR
- of childbearing potential but willing to practice effective contraception or abstinence during administration of the study drug and for 3 months after administration of the investigational study drug (dexpramipexole).
- Participation of Women:
- Contraception: Pre-clinical animal data demonstrated some fetal risk, suggesting there may a human reproductive risk. Subjects must agree not to become pregnant. Females of childbearing potential must have a pregnancy test before the first dose of dexpramipexole. Because of the risk involved, subjects and their partners must use two methods of birth control. They must continue to use both methods for 3 months after stopping the study drug. Two methods of birth control may be selected from the list included below:
- Hormonal contraception
- Male or female condoms with or without a spermicide
- Diaphragm or cervical cap with a spermicide
- Intrauterine device (IUD)
- If pregnancy is suspected or should occur, subjects must notify the study staff immediately.
Who cannot join
- A subject will not be eligible to participate in the study if any of the following conditions are fulfilled at the time of enrollment:
- Life-threatening HES or other condition that, in the Investigator s opinion, places the subject at undue risk by participating in the study
- Pregnant or breast-feeding
- History of malignancy, including solid tumors and hematologic malignancies (except basal cell and squamous cell cancers of the skin that have been completely excised and cured)
- HIV infection or any other known immunodeficiency.
- Biopsy-proven eosinophilic granulomatosis with polyangiitis
Show all 21Show fewer
- Positive test for FIP1L1/PDGFRA fusion gene
- Absolute neutrophil count <2000/microL at screening, or any documented history of neutropenia
- Renal dysfunction, defined as an estimated glomerular filtration rate (eGFR) of less than or equal to 80 mg/dL at screening (estimation of creatinine clearance using the MDRD formula).
- Cardiac abnormality defined as:
- Moderate to severely decreased cardiac function (left ventricular ejection fraction (LVEF) < 20% or history of LVEF <20% within the past 6 months or NYHA class IIIb or IV)
- History of angina or acute myocardial infarction in the past 6 months
- History or long QT syndrome or arrhythmia.
- A prolongation of QT/QTc interval (e.g., repeated demonstration of a QT/QTc interval >450 ms before study treatment administration) at screening, admission or pre-dose on Day 1.
- Any clinically important abnormalities in resting ECG that may interfere with the interpretation of QTc interval changes at screening, admission or pre-dose on Day 1.
- This includes subjects with any of the following:
- PR interval >210 ms;
- QRS >110 ms;
- Heart rate <45 bpm or >100 bpm (average of 3 assessments).
- Recent history or suspicion of drug or alcohol abuse in the preceding 6 months
- Treatment with an investigational drug in the previous 30 days
Trial locations and contacts
1 site in 1 country.
United States1 site
National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, 20892
About this study
Official title: An Open-Label, Proof-of-Concept Study to Evaluate the Safety and Efficacy of Dexpramipexole (KNS-760704) in Subjects With Hypereosinophilic Syndrome
Background:
- Eosinophils are white blood cells that fight infections. In people with hypereosinophilic syndrome (HES), eosinophil levels are too high and can damage their organs. HES is usually treated with steroids, but steroids can cause side effects and stop working over time. Researchers want to see if a drug called dexpramipexole, being developed by Knopp Pharmaceuticals, can help people with HES to reduce their steroid dose.
Read more
Objective:
- To test whether dexpramipexole can reduce the steroid dose needed to control eosinophilia and HES symptoms.
Eligibility:
- Adults 18 and older with HES who respond to steroids, but need more than 10 mg daily to control eosinophilia and symptoms.
Design:
- The study will last 9 months with 6 visits to NIH.
- Participants will be screened with medical history, physical exam, and urine and blood samples.
- Participants steroids will be tapered to the lowest effective dose. During this time, blood will be drawn weekly. Participants will take this dose for 2 weeks before starting the study drug.
- Participants will take the study drug twice daily by mouth for 12 weeks along with steroids. The steroid dose will not be decreased during this time and participants will be seen monthly for a medical history, physical examination and blood work.
- Just before and 12 weeks after starting the study drug, the following tests will be performed:
- medical history and physical exam
- blood and urine tests
- lung function tests
- electrocardiogram (measures heart electrical activity)
- echocardiogram (takes pictures of the heart using sound waves)
- bone marrow biopsy (a needle inserted into the hip bone that removes bone marrow cells for study)
- After 12 weeks, the participants steroid dose will be tapered again to the lowest effective dose while on study drug.
- Two weeks after the lowest effective dose is reached, participants will return for a medical history, physical examination, blood work, lung and heart tests.
- Participants who respond to the study drug may be able to continue to receive the drug on a planned separate study.
- Four weeks after stopping the study drug, participants will have medical history, physical exam, and blood tests.
Read the detailed description
Hypereosinophilic syndromes (HES) are a heterogeneous group of disorders characterized by peripheral eosinophilia and evidence of eosinophil-related end organ damage. Although a high proportion of patients respond initially to corticosteroid therapy, high doses are often necessary to control the eosinophilia and clinical symptoms, and many patients become relatively refractory to therapy and/or develop serious side effects.
Dexpramipexole (KNS 760704) is a synthetic aminobenzothiazole shown to safely reduce blood eosinophils counts in individuals with amyotrophic lateral sclerosis (ALS) in several clinical trials. The purpose of this proof-of- concept study is to evaluate the effect of dexpramipexole, an orally bioavailable small molecule, on circulating and tissue eosinophils in 10 subjects with HES. Following completion of eligibility assessments, subjects with absolute eosinophil count (AEC) < 1000/uL will enter a lead-in period, during which a standardized weekly corticosteroid taper will be undertaken to establish a "minimally effective corticosteroid dose" in each study subject. For subjects whose symptoms are stable but AEC > 1000/uL at the time of enrollment, the steroid dose at the time of enrollment will be defined as the minimally effective corticosteroid dose and no taper will be performed. Once the minimally effective corticosteroid dose is established, treatment with dexpramipexole 150 mg twice daily will begin. A standardized corticosteroid taper will begin after 12 weeks of treatment with dexpramipexole to determine the "minimally effective corticosteroid dose on dexpramipexole". Eosinophil counts and routine chemistries will be monitored weekly during corticosteroid tapering. End organ assessment, including echocardiogram, pulmonary function testing, and other studies as appropriate will be performed at study baseline, initiation of dexpramipexole therapy, 3 months after initial dosing with dexpramipexole, and the end of study visit. Bone marrow assessment will be performed prior to and after 12 weeks of dexpramipexole. Drug levels will be assessed prior to dexpramipexole and at week 12 and week 24 of dosing.
The primary efficacy endpoint will be the number of subjects with a greater than or equal to 50 % change in prednisone (or equivalent) dose to maintain absolute eosinophil count (AEC) at or below baseline (pre-enrollment) levels and control clinical symptoms (responder analysis). Assuming that 10 patients received study drug per protocol, at least 4 of them will need to meet this endpoint to significantly show (at the usual two-sided 5% level, equivalent to the onesided 2.5% level) that at least 10% of patients respond to dexpramipexole (exact binomial test). Safety will be assessed as the incidence of adverse events (AEs) (including serious adverse events [SAEs]), vital signs, clinical laboratory assessments, physical examination, electrocardiogram (ECG) tests, and body weight. Exploratory endpoints will include determination of the effect of dexpramipexole on measures of eosinophil activation, cytokine/chemokine profile and other immunologic parameters, and reduction of tissue eosinophils.
- Conditions
- Hypereosinophilic Syndrome
- Being tested
- Dexpramipexole
- Keywords
- Corticosteroids, Eosinophilia
- Medical subject headings
- Hypereosinophilic Syndrome, Eosinophilia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DexpramipexoleDrug
Dexpramipexole (KNS 760704) is a synthetic amino-benzothiazole developed for use in amyotrophic lateral sclerosis (ALS)
| Study group | Role | What they receive |
|---|---|---|
| Dexpramipexole | Experimental | Dexpramipexole treatment Receives: Drug: Dexpramipexole |
What is being measured
The primary outcome is the main question the study is designed to answer.
The corticosteroid dose after treatment with 150 mg twice daily of dexpramipexole as a percentage of the corticosteroid dose prior to treatment
Measured: After 3 months of taking dexpramipexole
The number of subjects with a greater than or equal to 50 % change in prednisone (or equivalent) dose to maintain absolute eosinophil count (AEC) at or below baseline (pre-enrollment)levels and control clinical symptoms
Measured: After 3 months of taking dexpramipexole
Secondary outcomes (3)
Reduction in circulating eosinophil and bone marrow eosinophil count after 3 months of treatment with dexpramipexole (prior to steroid taper)
Measured: After 3 months of dexpramipexole therapy
The number of subjects able to taper to <10 mg prednisone (or equivalent) dose to maintain absolute eosinophil count (AEC) at or below baseline (pre-enrollment) levels and control clinical symptoms
Measured: After 3 months of dexpramipexole therapy
Incidence and severity of adverse events
Measured: During dexpramipexole therapy
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Apr 2014 (estimated)
- Study start14 Mar 2014
- Primary completion30 Dec 2016
- Study completion23 Oct 2019 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Hypereosinophilic SyndromeStudies of the same condition, open and under way.
- More trials run by National Institute of Allergy and Infectious Diseases (NIAID)The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Sponsor type
- US National Institutes of Health
- Collaborators
- Knopp Biosciences
- Responsible party
- Sponsor
- Investigators
- Amy D Klion, M.D., Principal investigator, National Institute of Allergy and Infectious Diseases (NIAID)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02101138
- Sponsor's protocol ID
- 140063
- Other IDs
- 14-I-0063
- Status last verified
- 1 Dec 2017
- First submitted
- 14 Mar 2014
- Last updated
- 7 Dec 2017
- Results posted
- No
Publications
Ogbogu PU, Bochner BS, Butterfield JH, Gleich GJ, Huss-Marp J, Kahn JE, Leiferman KM, Nutman TB, Pfab F, Ring J, Rothenberg ME, Roufosse F, Sajous MH, Sheikh J, Simon D, Simon HU, Stein ML, Wardlaw A, Weller PF, Klion AD. Hypereosinophilic syndrome: a multicenter, retrospective analysis of clinical characteristics and response to therapy. J Allergy Clin Immunol. 2009 Dec;124(6):1319-25.e3. doi: 10.1016/j.jaci.2009.09.022.
PubMed 19910029Bozik ME, Mather JL, Kramer WG, Gribkoff VK, Ingersoll EW. Safety, tolerability, and pharmacokinetics of KNS-760704 (dexpramipexole) in healthy adult subjects. J Clin Pharmacol. 2011 Aug;51(8):1177-85. doi: 10.1177/0091270010379412. Epub 2010 Oct 19.
PubMed 20959524Cudkowicz ME, van den Berg LH, Shefner JM, Mitsumoto H, Mora JS, Ludolph A, Hardiman O, Bozik ME, Ingersoll EW, Archibald D, Meyers AL, Dong Y, Farwell WR, Kerr DA; EMPOWER investigators. Dexpramipexole versus placebo for patients with amyotrophic lateral sclerosis (EMPOWER): a randomised, double-blind, phase 3 trial. Lancet Neurol. 2013 Nov;12(11):1059-67. doi: 10.1016/S1474-4422(13)70221-7. Epub 2013 Sep 23.
PubMed 24067398Panch SR, Bozik ME, Brown T, Makiya M, Prussin C, Archibald DG, Hebrank GT, Sullivan M, Sun X, Wetzler L, Ware J, Fay MP, Dunbar CE, Dworetzky SI, Khoury P, Maric I, Klion AD. Dexpramipexole as an oral steroid-sparing agent in hypereosinophilic syndromes. Blood. 2018 Aug 2;132(5):501-509. doi: 10.1182/blood-2018-02-835330. Epub 2018 May 8.
PubMed 29739754
Study at a glance
From the registry record, last updated by the sponsor on 7 Dec 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label, Proof-of-Concept Study to Evaluate the Safety and Efficacy of Dexpramipexole (KNS-760704) in Subjects With Hypereosinophilic Syndrome
- ClinicalTrials.gov ID
- NCT02101138
- Acronym / sponsor ID
- 140063
- Conditions
- Hypereosinophilic Syndrome
- Keywords
- Corticosteroids, Eosinophilia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 15 participants (actual)
- Lead sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)US National Institutes of Health
- Collaborators
- Knopp Biosciences
- Study start
- 14 Mar 2014
- Primary completion
- 30 Dec 2016 (actual)
- Study completion
- 23 Oct 2019 (estimated)
- First posted
- 2 Apr 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Background:
- Eosinophils are white blood cells that fight infections. In people with hypereosinophilic syndrome (HES), eosinophil levels are too high and can damage their organs. HES is usually treated with steroids, but steroids can cause side effects and stop working over time. Researchers want to see if a drug called dexpramipexole, being developed by Knopp Pharmaceuticals, can help people with HES to reduce their steroid dose.
Objective:
- To test whether dexpramipexole can reduce the steroid dose needed to control eosinophilia and HES symptoms.
Eligibility:
- Adults 18 and older with HES who respond to steroids, but need more than 10 mg daily to control eosinophilia and symptoms.
Design:
- The study will last 9 months with 6 visits to NIH.
- Participants will be screened with medical history, physical exam, and urine and blood samples.
- Participants steroids will be tapered to the lowest effective dose. During this time, blood will be drawn weekly. Participants will take this dose for 2 weeks before starting the study drug.
- Participants will take the study drug twice daily by mouth for 12 weeks along with steroids. The steroid dose will not be decreased during this time and participants will be seen monthly for a medical history, physical examination and blood work.
- Just before and 12 weeks after starting the study drug, the following tests will be performed:
- medical history and physical exam
- blood and urine tests
- lung function tests
- electrocardiogram (measures heart electrical activity)
- echocardiogram (takes pictures of the heart using sound waves)
- bone marrow biopsy (a needle inserted into the hip bone that removes bone marrow cells for study)
- After 12 weeks, the participants steroid dose will be tapered again to the lowest effective dose while on study drug.
- Two weeks after the lowest effective dose is reached, participants will return for a medical history, physical examination, blood work, lung and heart tests.
- Participants who respond to the study drug may be able to continue to receive the drug on a planned separate study.
- Four weeks after stopping the study drug, participants will have medical history, physical exam, and blood tests.
Detailed description
Hypereosinophilic syndromes (HES) are a heterogeneous group of disorders characterized by peripheral eosinophilia and evidence of eosinophil-related end organ damage. Although a high proportion of patients respond initially to corticosteroid therapy, high doses are often necessary to control the eosinophilia and clinical symptoms, and many patients become relatively refractory to therapy and/or develop serious side effects.
Dexpramipexole (KNS 760704) is a synthetic aminobenzothiazole shown to safely reduce blood eosinophils counts in individuals with amyotrophic lateral sclerosis (ALS) in several clinical trials. The purpose of this proof-of- concept study is to evaluate the effect of dexpramipexole, an orally bioavailable small molecule, on circulating and tissue eosinophils in 10 subjects with HES. Following completion of eligibility assessments, subjects with absolute eosinophil count (AEC) < 1000/uL will enter a lead-in period, during which a standardized weekly corticosteroid taper will be undertaken to establish a "minimally effective corticosteroid dose" in each study subject. For subjects whose symptoms are stable but AEC > 1000/uL at the time of enrollment, the steroid dose at the time of enrollment will be defined as the minimally effective corticosteroid dose and no taper will be performed. Once the minimally effective corticosteroid dose is established, treatment with dexpramipexole 150 mg twice daily will begin. A standardized corticosteroid taper will begin after 12 weeks of treatment with dexpramipexole to determine the "minimally effective corticosteroid dose on dexpramipexole". Eosinophil counts and routine chemistries will be monitored weekly during corticosteroid tapering. End organ assessment, including echocardiogram, pulmonary function testing, and other studies as appropriate will be performed at study baseline, initiation of dexpramipexole therapy, 3 months after initial dosing with dexpramipexole, and the end of study visit. Bone marrow assessment will be performed prior to and after 12 weeks of dexpramipexole. Drug levels will be assessed prior to dexpramipexole and at week 12 and week 24 of dosing.
The primary efficacy endpoint will be the number of subjects with a greater than or equal to 50 % change in prednisone (or equivalent) dose to maintain absolute eosinophil count (AEC) at or below baseline (pre-enrollment) levels and control clinical symptoms (responder analysis). Assuming that 10 patients received study drug per protocol, at least 4 of them will need to meet this endpoint to significantly show (at the usual two-sided 5% level, equivalent to the onesided 2.5% level) that at least 10% of patients respond to dexpramipexole (exact binomial test). Safety will be assessed as the incidence of adverse events (AEs) (including serious adverse events [SAEs]), vital signs, clinical laboratory assessments, physical examination, electrocardiogram (ECG) tests, and body weight. Exploratory endpoints will include determination of the effect of dexpramipexole on measures of eosinophil activation, cytokine/chemokine profile and other immunologic parameters, and reduction of tissue eosinophils.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Dexpramipexole | Experimental | Drug: DexpramipexoleDexpramipexole treatment |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (16 listed)
- A subject will be eligible for participation in the study only if all of the following criteria apply:
- The subject is male or female, age greater than or equal to 18 years
- The subject has a documented history of HES requiring greater than or equal to 10 mg prednisone (or equivalent) to maintain disease control.
- HES is defined as 1) peripheral blood eosinophilia (>1500 eosinophils/microL) on at least two occasions, 2) signs and symptoms of organ system involvement attributable to the eosinophilia, and 3) exclusion of secondary causes of eosinophilia, such as parasitic helminth infection, drug hypersensitivity and neoplasms, for which appropriate therapy is directed at the underlying cause
- HES symptoms are stable on the current corticosteroid dose.
Key exclusion criteria (21 listed)
- A subject will not be eligible to participate in the study if any of the following conditions are fulfilled at the time of enrollment:
- Life-threatening HES or other condition that, in the Investigator s opinion, places the subject at undue risk by participating in the study
- Pregnant or breast-feeding
- History of malignancy, including solid tumors and hematologic malignancies (except basal cell and squamous cell cancers of the skin that have been completely excised and cured)
- HIV infection or any other known immunodeficiency.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| DexpramipexoleDexpramipexole (KNS 760704) is a synthetic amino-benzothiazole developed for use in amyotrophic lateral sclerosis (ALS) | Drug | Dexpramipexole |
Primary outcome
The corticosteroid dose after treatment with 150 mg twice daily of dexpramipexole as a percentage of the corticosteroid dose prior to treatment
Time frame: After 3 months of taking dexpramipexole
The number of subjects with a greater than or equal to 50 % change in prednisone (or equivalent) dose to maintain absolute eosinophil count (AEC) at or below baseline (pre-enrollment)levels and control clinical symptoms
Time frame: After 3 months of taking dexpramipexole
3 secondary outcomes
Reduction in circulating eosinophil and bone marrow eosinophil count after 3 months of treatment with dexpramipexole (prior to steroid taper)
Time frame: After 3 months of dexpramipexole therapy
The number of subjects able to taper to <10 mg prednisone (or equivalent) dose to maintain absolute eosinophil count (AEC) at or below baseline (pre-enrollment) levels and control clinical symptoms
Time frame: After 3 months of dexpramipexole therapy
Incidence and severity of adverse events
Time frame: During dexpramipexole therapy
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Dec 2017.
- Background: PubMed 19910029Ogbogu PU, Bochner BS, Butterfield JH, Gleich GJ, Huss-Marp J, Kahn JE, Leiferman KM, Nutman TB, Pfab F, Ring J, Rothenberg ME, Roufosse F, Sajous MH, Sheikh J, Simon D, Simon HU, Stein ML, Wardlaw A, Weller PF, Klion AD. Hypereosinophilic syndrome: a multicenter, retrospective analysis of clinical characteristics and response to therapy. J Allergy Clin Immunol. 2009 Dec;124(6):1319-25.e3. doi: 10.1016/j.jaci.2009.09.022.
- Background: PubMed 20959524Bozik ME, Mather JL, Kramer WG, Gribkoff VK, Ingersoll EW. Safety, tolerability, and pharmacokinetics of KNS-760704 (dexpramipexole) in healthy adult subjects. J Clin Pharmacol. 2011 Aug;51(8):1177-85. doi: 10.1177/0091270010379412. Epub 2010 Oct 19.
- Background: PubMed 24067398Cudkowicz ME, van den Berg LH, Shefner JM, Mitsumoto H, Mora JS, Ludolph A, Hardiman O, Bozik ME, Ingersoll EW, Archibald D, Meyers AL, Dong Y, Farwell WR, Kerr DA; EMPOWER investigators. Dexpramipexole versus placebo for patients with amyotrophic lateral sclerosis (EMPOWER): a randomised, double-blind, phase 3 trial. Lancet Neurol. 2013 Nov;12(11):1059-67. doi: 10.1016/S1474-4422(13)70221-7. Epub 2013 Sep 23.
- Derived from this study: PubMed 29739754Panch SR, Bozik ME, Brown T, Makiya M, Prussin C, Archibald DG, Hebrank GT, Sullivan M, Sun X, Wetzler L, Ware J, Fay MP, Dunbar CE, Dworetzky SI, Khoury P, Maric I, Klion AD. Dexpramipexole as an oral steroid-sparing agent in hypereosinophilic syndromes. Blood. 2018 Aug 2;132(5):501-509. doi: 10.1182/blood-2018-02-835330. Epub 2018 May 8.
- NIH Clinical Center Detailed Web PageLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Amy D Klion, M.D. principal investigator
National Institute of Allergy and Infectious Diseases (NIAID)
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Dexpramipexole in people with Hypereosinophilic Syndrome, run by National Institute of Allergy and Infectious Diseases (NIAID) at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 16 inclusion and 21 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 7 Dec 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The corticosteroid dose after treatment with 150 mg twice daily of dexpramipexole as a percentage of the corticosteroid dose prior to treatment (time frame: After 3 months of taking dexpramipexole). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02101138, which also lists 4 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Dec 2017.